Ocuphire Pharma’s oral diabetic retinopathy drug missed the primary endpoint of an improvement in patient scores on the diabetic retinopathy severity scale.
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Ocuphire Pharma will move forward with an FDA end-of-Phase II meeting despite missing the primary endpoint with its oral diabetic retinopathy (DR) drug, APX3330.
The primary endpoint assessed whether APX3330 could demonstrate an improvement in patient scores on the diabetic retinopathy severity scale (DRSS), a categorical tool that evaluates the severity of retinopathy in the eyes by evaluating photographs.
APX3330 did not show a statistically significant improvement in reversing the impacts of diabetic retinopathy on vision, according to the data readout.
The candidate did achieve statistical significance in preventing clinically meaningful progression of DR after 24 weeks of treatment in both eyes. This was a key pre-specified secondary endpoint.
Additionally, visual acuity was stable in those treated with the therapeutic with fewer patients losing distance vision than those treated with placebo.
APX3330 also demonstrated a favorable safety and tolerability profile. Treatment-related adverse events were uncommon, with most being mild in severity, according to the press release.
Phase III Objectives
During a conference call Wednesday, the company stated that, if approved, APX3330 could be an important primary preventative therapeutic option for patients at an earlier stage of disease.
“It’s an obvious clinical intervention,” said Charles Wykoff, M.D., Ph.D., a vitreoretinal specialist with the Retina Consultants of Texas, during the presentation. Clinicians are looking for a systemic, safe drug that can help prevent patients from losing their eyesight, he said. This is an outcome for many patients with diabetic retinopathy.
Ocuphire noted in its presentation that prevention of 3-step binocular worsening in DRSS scores in both eyes is a suitable endpoint for a Phase III trial.
“There has been an opportunity for retinal drugs to be approved based on either the improvement or prevention of worsening of retinal disease,” said Mitchell Brigell, Ph.D., head of clinical development and strategy.
A Phase III trial will also highlight the importance of evaluating DRSS change in both eyes, Ocuphire noted in the presentation. This endpoint is important because APX3330 is a systemic drug. Other approved drugs, such as Anti-VEGF medications, are usually injected into one eye at a time.
The Oral Drug Difference
Although there are drugs approved that effectively slow the progression of and reverse the effects of DR, they are not widely used, Wykoff said.
“The biggest challenge is the chronic delivery of an intravitreal injection,” he said.
Intravitreal injections are delivered directly into the vitreous cavity in the back of the eye. As many patients are well-sighted when diagnosed, this can prevent them from seeking treatment to slow progression, he said.
Further analysis of the trial data will provide insights for Phase III registration and trial design, according to Ocuphire’s presentation.
Advance development for APX3330, including the cGMP drug, NDA-enabling work, the first Phase III trial and regional partnerships, is fully funded into 2025.
